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Cancer drug may help treat advanced Parkinson’s disease and Lewy body dementia

November 12, 2015
by Nicole Stempak, Associate Editor
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A drug used to treat leukemia was found to improve cognition, motor skills and non-motor function for people with Parkinson’s disease with dementia and Lewy body dementia, according to a study.

Fernando Pagan, MD, associate professor of neurology at Georgetown University Medical Center and director of the Movement Disorders Program at MedStar Georgetown University Hospital presented the findings at Neuroscience, the annual meeting of the Society for Neuroscience.

“To my knowledge, this study represents the first time a therapy appears to reverse—to a greater or lesser degree depending on stage of disease—cognitive and motor decline in patients with these neurodegenerative disorders,” says Pagan in a university-issued press release. “But it is critical to conduct larger and more comprehensive studies before determining the drug’s true impact,” he cautioned.

Investigators reported that the six-month, dose-escalating study of nilotinib (Tiasigna provided by Novartis), from 150 to 300 mg daily, a treatment for chronic myelogenous leukemia produced benefit for all study participants who completed the trial. Ten out of 12 participants reported meaningful clinical improvements. Participants also showed positive changes in relevant cerebrospinal fluid biomarkers of Parkinson’s with statistically significant changes in total Tau and p-Tau, which increase with the onset of dementia.

In addition, researchers found that the drug penetrates the blolod-brain barrier in amounts greater than dopaminie drugs.

“When used in higher doses for CML, nilotinib forces cancer cells into autophagy—a biological process that leads to the death of tumor cells. The dose used in CML treatment is significantly higher than what we used in our Parkinson’s study,” says co-author Charbel Moussa, MD, PHD and director of Georgetown’s Laboratory of Dementia and Parkinsonism. “It appears that in smaller doses once a day, nilotinib turns on auotphagy for about four to eight hours – long enough to clean out the cells without causing cell death. Then proteins that build up again will be cleared when the drug is given again the next day.”

Researchers caution interpreting phase 1 results because the study was designed to test safety, not efficacy, and lacked a control group. Researchers are planning a double-blind, placebo-controlled phase 2 clinical trial testing nilotinib in people with Parkinson’s disease and other neurodegenerative diseases, including Alzheimer’s disease. The study is expected to begin in 2016.

Read the news release here.

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